NCT02563860

Brief Summary

This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jul 2015

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2015

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

July 20, 2015

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 30, 2015

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

May 7, 2019

Completed
Last Updated

July 12, 2019

Status Verified

July 1, 2019

Enrollment Period

1 year

First QC Date

July 20, 2015

Results QC Date

May 19, 2018

Last Update Submit

July 11, 2019

Conditions

Rett Syndrome

Keywords

Treatment trialLovastatin

Outcome Measures

Primary Outcomes (1)

  • Gait Velocity as Measured by GAITRite System

    To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)

    During final week of treatment, week 32

Secondary Outcomes (1)

  • Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System

    final week of treatment, Week 32

Study Arms (1)

Open label

EXPERIMENTAL

Treatment with Lovastatin, dose escalating trial according to the following schedule: 10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.

Drug: Lovastatin

Interventions

LovastatinDRUG

dose escallating

Also known as: Mevacor
Open label

Eligibility Criteria

Age3 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Females patients,
  • Genetically confirmed RTT,
  • Ambulatory.

You may not qualify if:

  • Presence of co morbid non-Rett related disease,
  • History of adverse reaction/hypersensitivity to statins,
  • Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,
  • Active liver disease,
  • Concomitant use of strong CYP3A4 inhibitors,
  • Condition predis-posing to renal failure secondary to rhabdomyolysis (scoliosis surgery within 6 months),
  • Oral contraceptives use.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Montefiore Medical center

The Bronx, New York, 10467, United States

Location

MeSH Terms

Conditions

Rett Syndrome

Interventions

Lovastatin

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

NaphthalenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic ChemicalsPolycyclic Compounds

Results Point of Contact

Title
Aleksandra Djukic
Organization
Montefiore MC
adjukic@montefiore.org
7189204378

Study Officials

  • Aleksandra Djukic, MD PhD

    Montefiore Medical Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Clinical Neurology, Director, Tri State Rett Syndrome Center

Study Record Dates

First Submitted

July 20, 2015

First Posted

September 30, 2015

Study Start

July 1, 2015

Primary Completion

July 1, 2016

Study Completion

July 1, 2016

Last Updated

July 12, 2019

Results First Posted

May 7, 2019

Record last verified: 2019-07

Data Sharing

IPD Sharing
Will not share

Locations

US(1)